The US Food and Drug Administration (FDA) has accepted Roche’s biologics licence application (BLA) and granted priority review for emicizumab prophylaxis (preventative) to treat patients with haemophilia A with factor VIII inhibitors.
Haemophilia A is an inherited, serious condition in which the patient’s blood fails to clot properly, thereby leading to uncontrolled and often spontaneous bleeding.
Created by Chugai Pharmaceutical, emicizumab is currently being developed by the company in collaboration with Roche and Genentech.
Emicizumab is an investigational bispecific monoclonal antibody that has been developed to bring IXa and X factors together.
IXa and X are proteins essential in activating the natural coagulation cascade and are used to restore the blood clotting process.
The therapy is a subcutaneous treatment that can be administered once every week on adults, adolescents and children with haemophilia A with factor VIII inhibitors, which reduces treatment options and enhances the risk of life-threatening bleeds and repeated bleeds that result in long-term damage.
Roche’s BLA for emicizumab is based on data from Phase III HAVEN 1 study carried out on patients of 12 years of age and older, in addition to interim results from the Phase III HAVEN 2 trial involving children younger than 12.
Roche chief medical officer and global product development head Dr Sandra Horning said: “Results of our Phase III study in adults and adolescents, as well as early Phase III results in children, showed that emicizumab has significant potential to help people with haemophilia A with inhibitors who face major challenges in preventing and treating bleeds.
“We are working with the FDA to hopefully bring this new prophylactic treatment option to the haemophilia A inhibitor community as soon as possible.”
The US agency is expected to take a decision on the BLA by 23 February next year.